Clinical Trial Details

NCT ID: NCT02595489
Date Last Changed: October 12, 2016


Research Study Summary

A Phase 1/Phase 2 clinical study for patients with X-linked Adrenoleukodystrophy

Research Study Title

A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy


In this pilot study, the investigators will assess the safety of two high-dose regimens of oral vitamin D supplementation and measure the effects of vitamin D supplementation on markers of oxidative stress and inflammation in the blood and brain of study participants before, during, and after taking vitamin D supplements.

The goal of the study is to establish research measures (i.e. biomarkers) and an optimal dose for vitamin D supplementation in boys with the X-linked adrenoleukodystrophy (ALD) genotype.

To Learn more

Recruitment Details

3 to 12 Years
Overall Status
Lead Sponsor
Stanford University
36 Months
Facility Type


Male ages 3 Years to 12 Years

Criteria for enrollment to screening:

  1. Molecular confirmation of X-linked ALD (VLCFA elevation & ABCD1 mutation)

  2. Male

  3. Age 3yrs - 12yrs at screening

Criteria for assignment to drug:

  1. Plasma 25-hydroxy vitamin D level ≤ 35ng/ml in past 90 days

  2. MRI brain in past 30 days that is negative for evidence of active cerebral demyelination

Exclusion Criteria:

  • history of liver or kidney disease

  • history of nephrolithiasis

  • history of hyperthyroidism

  • history of ulcerative colitis, Crohn's disease, celiac disease

  • taking medication interfering with gastrointestinal absorption

  • contraindication or inability to complete MRI every 6 months

Site Locations (1)

Country State City Zip Facility and Contact
United States California Palo Alto 94304 Stanford University
Keith Van Haren, MD

Keith Van Haren, MD
Principal Investigator


Keith Van Haren, MD


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